Table IV.

Treatments with reported benefit in mitochondrial myopathy which may benefit from further study in blinded placebo-controlled trials.

Agent	Reported benefit	Evidence
Ascorbate and menadione	Improvement on 31PNMR and symptomatic	Single case reports in complex III deficiency (92,93)
High-fat diet with vitamins and CoQ10	Short-term improvement in neurodevelopment, seizure control, level of consciousness	Open-label study of 15 paediatric patients(94)
Idebenone	Biochemical improvements; delayed disease progression; improvement of respiratory function	Single case reports(53–55)
L-arginine	Reduction in acute symptoms of stroke-like episodes, reduction of incidence of stroke-like episodes in MELAS; improvement of TCA metabolic rate on C11-PET in cardiomyopathy.	Non-blinded, placebo-controlled study of 24 MELAS patients(64); C11-PET study in 6 patients with MELAS(65)
Magnesium	Resolution of refractory status epilepticus in Alpers syndrome	Two patients(66)
Nicotinamide	Biochemical improvements. Reduced encephalopathy and stroke-like episodes in one case	Six-month open label trial of seven MELAS patients(95), and single case reports(96,97)
Succinate	Improvement of respiratory muscle weakness; decrease in stroke-like episodes.	Single case reports(98,99)